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Reaching for a Cure

Reaching for a Cure. Summer 2007 (Group 14) Group Members: Jessica Barnard William Goode Julienne Kung Philippe Lacasse Sarah Pettyjohn. Reaching for Knowledge. Genetic disorders are the result of deleterious mutations in the human genome.

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Reaching for a Cure

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  1. Reaching for a Cure Summer 2007 (Group 14) Group Members: Jessica Barnard William Goode Julienne Kung Philippe Lacasse Sarah Pettyjohn

  2. Reaching for Knowledge • Genetic disorders are the result of deleterious mutations in the human genome. • They cause the synthesis of non-functional or less efficient proteins. • Genetic disorders are caused by the person’s genes where genetic predisposition increases the probability of a disease occurring. • The traditional approach was to treat the symptoms. A modern trend has focused on the source of the problem, the genes themselves.

  3. Today: Cure by Prevention • Prenatal Diagnosis • Microarrays (“Gene Chips”) • Donor Gametes/Embryos • Anonymous Donor Screening • Sperm Separation • Preimplantation Genetic Diagnosis • Polar Body Biopsy • Blastocyst Biopsy

  4. Tomorrow: Moderating Nature • Influencing the Central Dogma • Drugs • Environmental Control

  5. Tomorrow: Modifying Nature • Germline Gene Therapy • General Overview • Insertion of Artificial Chromosomes • Ooplasmic Transfer

  6. Now: Adjusting Life • Use drugs to suppress effects of dysfunction. • Remove affected cells if possible, otherwise… • Make the patient as comfortable as possible by treating symptoms as much as possible. • Consider the balance of longevity and standard of living.

  7. Later: Editing Genes • Gene Therapy • Viral Insertion • Chemical Insertion (“Naked DNA”) • Extraction of Somatic Cells, Genetic Engineering and Re-introduction

  8. Later: Altering Body Composition • Stem Cells • Repopulating areas deficient in certain cells. • Oral Therapies • Injection Therapies • Cell Grafts • Moving healthy cells where they are needed.

  9. Grasping Knowledge • As for prenatal treatment, the most effective method of cure is prevention. Most options include careful family planning. • After the individual is born, medicine will eliminate the disease if possible, but usually, we simply treat symptoms. • In the future, we hope to be able to adjust the human genome to eradicate diseases entirely.

  10. Works Cited • "DNA Chip Technology." DNA Microchip Technology. 8 May 2007. The National Human Genome Research Institute. 11 July 2007 <http://www.genome.gov/10000205>. • "Gene Therapy for Cancer: Questions and Answers." National Cancer Institute Factsheet. 31 Aug. 2006. National Cancer Institute. 12 July 2007 <http://www.cancer.gov/cancertopics/factsheet/Therapy/gene>. • "NINDS Huntington's Disease Information Page." 13 Feb. 2007. 11 July 2007 <http://www.ninds.nih.gov/disorders/huntington/huntington.htm> • "NINDS Muscular Dystrophy Information Page." 13 Feb. 2007. 11 July 2007 <http://www.ninds.nih.gov/disorders/md/md.htm> • “Gene Therapy”. Oxford Illustrated Companion to Medicine . (2001): 334. Ebscohost . 3 July 2007. • “Germline Gene Therapy.” UCLA Program on Science Technology and Society. 20 Mar. 1998. 10 July 2007 <http://www.ess.ucla.edu/huge/germdef.html>. • Cohen, Jacques et al. “Ooplasmic transfer in mature human oocytes” Molecular Human Reproduction vol. 4 no. 3 <http://molehr.oxfordjournals.org/cgi/reprint/4/3/269.pdf> • De Wolfe, Erick. Gene Therapy: A new generation of molecular medicine 5 July 2007 <http://www.cc.ndsu.nodak.edu/instruct/mcclean/plsc431/students/dewold.htm>. • Edwards, D. “Future ‘Patchwork’ Cure for Hemophilia?” Science News . 131 (1987): 168-167. Ebscohost . 3 July 2007. • Genetic Disorders: PTC 124. PTC Therapeutics. 5 July 2007 <http://www.ptcbio.com/3.1.1_generic_disorders.aspx> • Jones, Dan. "Superimmune." New Scientist (2007): 42-45. ISI Web of Knowledge. Georgia Tech Library. 1 July 2007. • Jones, Peter. “Towards a Cure.” Living with Haemophilia . (2002): 253-262. Ebscohost . 3 July 2007. • Jones, Shirley L., and Lee A. Fallon. "Reproductive Options for Individuals At Risk for Trasmission of a Genetic Disorder." Jognn: Clinical Issues 31 (2002): 193-199. ISI Web of Knowledge. Georgia Tech Library. 1 July 2007. • Jonietz, Erika. “Chromosome Cure”. Technology Review . 106 (2003): 29. Ebscohost . 3 July 2007. • Peter, Kennedy. "Potential Use of Herpes Simplex Virus (HSV) Vectors for Gene Therapy of Nurological Disorders." Brain (1997): 1245-1259. ISI Web of Knowledge. Georgia Tech Library. • Silverstein, Alvin; et al. “Chapter Six: Cells and the Future”. Cells . (2002): 47. Ebscohost . 3 July 2007. • Thompson, James S. Genetics in Medicine 3rd ed. Philadelphia: Saunders, 1980

  11. Images Cited Slide 1 • http://www2.carthage.edu/~pfaffle/hgp/genome.jpeg Slide 2 • http://nanopedia.case.edu/image/dna.jpg Slide 3 • http://www.yorku.ca/kdenning/images/bioanthropology/DNAKit_illustration_image.jpg • http://keck.med.yale.edu/affymetrix/technology.htm Slide 4 • http://www.ptcbio.com/3.1.1_generic_disorders.aspx • http://www.cnn.com/SPECIALS/2005/cnn.25/interactive/gallery.medical/1.genome.jpg Slide 5 • http://www.sgeier.net/fractals/flam3/fractals/DNA.jpg • http://www.em1.molmed.uni-erlangen.de/AGSchneider/JEB.html Slide 6 • www.hgsc.bcm.tmc.edu/graphics/genome3.jpg Slide 7 • http://www.esa.int/images/CHROMOSOME400.JPG • http://ghr.nlm.nih.gov/handbook/illustrations/therapyvector Slide 8 • http://www.csa.com/discoveryguides/stemcell/overview.php • http://www.mcdonald.k12.oh.us/home/students/07dugad/Biology%20Project/chromosome_3d.gif Slide 9 • http://digitalartmuseum.com/october/billingsley/artwork/DNA-synthesis.billingsley.jpg Slide 10 • http://english.pravda.ru/img/idb/21-human_genes.jpg Slide 11 • http://archives.cnn.com/2000/HEALTH/06/27/genome.01/story.human.genome.3.jpg

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